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Promising interim Phase 3 data spurs epidermolysis bullosa trial expansion for Amryt

Published on 07/01/19 at 10:47am

Rare disease firm Amryt has announced interim efficacy data for its epidermolysis bullosa (EB) therapy AP101, showing that things are looking good so far for further development of the treatment.

The Phase 3 trial investigating the drug – the largest ever conducted in EB – was evaluated by an Independent Data Monitoring Committee (IDMC). After examining unblinded efficacy data gathered to assess the primary endpoint over the course of the first half of the trial, the IDMC determined that the study should continue and increase its total number of participants from 48 to 230, in order to achieve 80% statistical power.

AP101 was originally accepted for review by the FDA in September last year. EB is a rare, life-limiting genetic condition in which a patient is born without the necessary protein anchors between the outer and inner layers of their skin, leading it to tear and blister with painful sores. Around 500,000 people around the world are estimated to be affected, while AP101 is projected to have a market potential of over €1 billion.

“The IDMC’s recommendation is good news for Amryt as it allows us to continue the trial with only a modest increase in the size of the study,” remarked Joe Wiley, CEO of Amryt Pharma. “This brings us closer to potentially delivering a treatment for patients with EB.  We would like to thank sincerely the patients, families and clinical investigators involved in the EASE trial to date. Today’s news is a significant milestone for Amryt and our shareholders in building towards our vision of becoming a global leader in rare and orphan diseases.”

Amryt confirmed that it plans to release full top-line data from the study in the second half of the year, with the recruitment beginning shortly for the expanded study.

Matt Fellows

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